Grants Search Results

Langerhans cell histiocytosis (LCH) is a rare cancer of the immune cells that can cause a wide range of symptoms, ranging from a rash to lethal multi-organ disease. Dr. Zinn hypothesizes that a patient's symptoms are determined by a combination of the patient's specific mutation and the specific immune cell that becomes mutated. Dr. Zinn is investigating the causes of LCH in order to develop the most effective and safe therapies for each patient.

Targeted therapy works by attacking an abnormal gene product that is specific to the cancer type. Only a minority of neuroblastoma types show genetic drivers, which makes it difficult to develop targeted therapy. Most neuroblastomas show too many or too few copies of large chromosomal regions, called CNAs. Dr. Weiss is studying the connection between CNAs and neuroblastoma, to determine if it CNA is a possible candidate for targeted therapy. Dr. Weiss is engineering CNAs to create CNA-driven models of neuroblastoma, which he will then use to identify CNA-specific therapies to treat neuroblastoma.

Diffuse Intrinsic Pontine Glioma is a type of incurable brain tumor that affects young children. Despite treatment with radiation and chemotherapy, the tumor exhibits resistance to current treatments and grows back. Dr. Beroukhim is studying the tumors at a single-cell level to determine how they become resistant to treatments, which will help guide the development of combination therapies to improve outcomes. This grant is made with generous support from the McKenna Claire Foundation established by the Wetzel family in memory of their daughter, McKenna. Their mission is to cure pediatric brain cancer by raising awareness, increasing community involvement and funding research.

Treatment of childhood acute lymphoblastic leukemia (ALL) using chemoradiation can be successful, but it is difficult to manage treatment-associated side events and secondary cancers. Furthermore, in relapsed/refractory patients, the overall prognosis remains dismal. Direct inhibition of the main proteins promoting cancer (the “oncogenes”) is not successful in ALL. Dr. Ntziachristos's "Just Do It…...and be done with it" St. Baldrick'’s Research Grant will study certain oncogene-supporting mechanisms that might be specific to a diseased state, and not to a healthy state. Dr. Ntziachristos has selected one of these mechanisms to target in ALL models, and is assessing the anti-cancer activity that results. Such experiments could pave the way for clinical trials for high-risk disease. This grant is named for the “"Just Do It...…and be done with it"” Hero Fund created in honor of Sara Martorano who doesn'’t let anything dim her sparkle and has a compassionate heart and smile. It also celebrates the courage of all cancer kids through treatment and the support of their family and friends.

The interaction between two important cancer-related proteins called MDM4 and TOP2A may cause cancer and contribute to disease progression. Dr. Zhou is studying the regulation of MDM4 and TOP2A to identify small-molecule inhibitors (agents) that can block the MDM4-TOP2A interaction, leading to inhibition of these two proteins. The results of these studies will provide important clues to help scientists develop novel methods and drugs to specifically and simultaneously target TOP2A and MDM4 for treatment of pediatric cancer patients.

Childhood brain tumors are frequently quite different than those of adults. Dr. Eck's For the Love of Jack St. Baldrick'’s Research Grant aims to find new “targeted” therapies for low-grade astrocytomas (a type of brain tumor) in children that are caused by a mutation in a protein called BRAF. BRAF mutations are common in cancer, and drugs have been developed that are effective in some tumors caused by one type of BRAF mutation. Unfortunately, these drugs do not work on the BRAF mutation found most often in pediatric brain tumors. Dr. Eck is using detailed information about the molecular structure of the BRAF mutation found in pediatric brain tumors to discover new drugs that specifically target this cause of brain tumors in children. Jack Tweedy was diagnosed with brain and spinal cancer when he was two. Since then he has endured multiple surgeries and 270 weeks of chemotherapy but never fails to uplift those around him. Together with his family, he inspires others to help fund the best research to ensure that all cancer warriors have better treatment options.

Genes instruct cells to do their jobs through making specific proteins. In the human body, all cells store these "instructions" in the chromosomes. When chromosomes break off, the broken pieces sometimes change places and create new chromosomes. These changes are called chromosomal translocations. Dr. Su is studying how chromosomal translocations cause deadly diseases in children and young adults, and more importantly, is investigating possible clinical options to correct these abnormal conditions.

Osteosarcoma is the most common bone cancer in children. Typical treatment includes cancer-killing drugs for several weeks followed by surgery. These drugs work for some patients but not for others. Doctors need a way to identify which patients respond to treatment and which don’t. Dr. Roblyer is studying the efficacy of a new light-based technology to determine when and if patients respond to treatment. This technology is low-cost, fast, and measurements are taken with a hand-held or wearable probe, like a Fitbit for cancer. If successful, this research will provide doctors with a new and simple method to personalize and improve treatment for each child with osteosarcoma.

Progress in improving therapy for Acute myeloid leukemia (AML) has been slow and survival rates for patients remain quite low. Thus, there is a great need for more effective and less toxic therapies for AML. Dr. DeGregori has identified a new molecule called MCJ, the loss of which is associated with the resistance of cancers to therapies. Dr. DeGregori has developed novel drugs that can restore MCJ function in cancer cells, and is investigating whether these MCJ activating drugs can be used improve therapies for AML.

Children's cancer can spread through the body by hiding from the bodys immune system. There are certain cells, called regulatory T cells, that make it easier for cancer to hide by turning down the bodys immune system. Children with cancer who have a higher number of these cells seem to have a poorer outcome. Additionally, some cancers have these cells inside them. Dr. Mathias's lab has made a drug that can kill regulatory T cells, and will test this drug in a model to see the effects of killing the regulatory T cells, hopefully helping the body to improve the immune system's ability to kill cancer cells.

Based on progress to date, Dr. Deel was awarded a new grant in 2018 to fund an additional year of this Fellow award. Rhabdomyosarcoma is a childhood cancer of the muscle that has two major subtypes. Children with the alveolar type do very poorly, as these are frequently recurrent and metastatic. Alveolar rhabdomyosarcoma cells have a specific fusion protein that is a powerful cancer driver and that does not yet have a pharmaceutical treatment. Dr. Deel and his team recently found that the Hippo pathway, normally functions to suppress tumor growth, is not correctly regulated in alveolar rhabdomyosarcoma, which leads to TAZ (a co-activator) activating pro-tumoric gene transcription. As the Aiden's Army St. Baldrick's Fellow, Dr. Deel is studying the interaction between the protein and the co-activator TAZ as a novel therapeutic target. Aiden Binkley was diagnosed with Stage IV rhabdomyosarcoma at age 8. This bright, funny and courageous little boy believed he got cancer so he could grow up to find a cure for it. His vision is being carried on by Aiden’s Army through the funding of research. They will march until there is a cure!

Neuroblastoma is a common type of childhood cancer that arises from nerve-like cells and causes tumors just outside the brain. Dr. George is studying tumor cells from patients to discover new molecular targets for treatment of neuroblastoma. Dr. George and his team are growing tumor cells from patients and examining their electrical properties in order to identify specific proteins that carry electrical current that may be new targets for treating neuroblastoma using existing non-cancer drugs.

Relatively little is currently known about the cellular and molecular mechanisms of medulloblastoma spread throughout the spinal fluid. Dr. Keller's preliminary findings show that medulloblastoma tumor cells actively migrate to the surface of the covering of the brain and spine, rather than arriving passively. Dr. Keller is investigating the efficacy of stopping the tumor cells from migrating to the brain and spine surfaces, which, if successful, will provide a rationale for future clinical trials.

Based on progress to date, Dr. John was awarded a new grant in 2018 to fund an additional year of this Fellow award. Unfortunately, even with intensive treatment, only 60% of children with acute myeloid leukemia (AML) will survive the disease. Intensifying standard therapies have failed to improve survival rates, so a new approach is needed. Dr. John is creating a novel receptor that will specifically target a marker on AML cells. He will put this new receptor on T-cells of the body, which will then target and kill the leukemia cells. Dr. John hopes to improve outcomes for pediatric AML with this grant. This grant is generously supported by Super Soph's Pediatric Cancer Research Fund. Sophie Rossi was diagnosed with AML at 3 months of age. Throughout her courageous battle, she was always smiling, always joyful. This fund was created to honor her spunky, sweet spirit by funding research to find cures for AML and all childhood cancers.

Genetic studies have identified the molecular causes of childhood cancers such as T-cell leukemia and neuroblastoma. A recurrent theme in these cancers is abnormal activation of the MYC gene. Accordingly, researchers like Dr. Wendel have spent much time and effort in trying to identify inhibitors of MYC as they believe these could be very powerful therapies for these childhood cancers. Dr. Wendel and his colleagues recently found a new way to block the production of MYC using a natural compound. The natural product is rare and hard to come by and therefore Dr. Wendel and his colleagues are exploring ways to generate synthetic drugs based on this plant product. With support from the St. Baldrick’s Foundation they are working to bring this new strategy to the clinic. They focus especially on heavily pre-treated and relapsed childhood leukemia because affected children have few options and they hope to make a difference.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Pediatric Blood & Marrow Transplant Consortium. For a description of this project, see the consortium grant made to the lead institution: the National Marrow Donor Program, Minneapolis, MN.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Pediatric Blood & Marrow Transplant Consortium. For a description of this project, see the consortium grant made to the lead institution: the National Marrow Donor Program, Minneapolis, MN.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Pediatric Blood & Marrow Transplant Consortium. For a description of this project, see the consortium grant made to the lead institution: the National Marrow Donor Program, Minneapolis, MN.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Pediatric Blood & Marrow Transplant Consortium. For a description of this project, see the consortium grant made to the lead institution: the National Marrow Donor Program, Minneapolis, MN.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Pediatric Blood & Marrow Transplant Consortium. For a description of this project, see the consortium grant made to the lead institution: the National Marrow Donor Program, Minneapolis, MN.